enabling transformational impact

recent breakthroughs in stem cell biology together with technological advances for
phenotypic, functional screening have unlocked our ability to harness the potential of
adult stem- and precursor cells. our novel drug discovery approach is based on the
concept of selective activation of endogenous progenitor cells for controlled tissue
repair and regeneration by small molecules.

news

endogena presents at JLABS Toronto

At the highly attended two year anniversary of JLABS Toronto on May
10th, our VP discovery biology, Daphna Mokady, explained endogena's platform approach
for endogenous regeneration.

enable

endogena therapeutics is a pioneer in the discovery and development of first-in-class
endogenous regenerative medicines.

nobel laureate Shinya Yamanaka’s breakthrough discovery that mature cells can be transformed
into stem cells has opened the way to the therapeutic potential of regulating endogenous
stem cells by small molecule medicines. harnessing this knowledge opens the door to new
potential in addressing unmet medical needs associated with degenerative diseases, including
genetic diseases and those resulting from ageing.

our transformational drug discovery paradigm is based on the concept of selective activation
of endogenous progenitor cells for controlled tissue repair and regeneration.

endogena uses artificial intelligence to elucidate the links between structures of small
molecules and their effect on lineage-specific differentiation pathways - our platform
enables the identification of molecular modulators of progenitor cell regeneration, specific
to tissues and organs.

in our lead program, we have identified ways in which neuroectoderm pathway
retinal progenitor cells can be targeted by small-molecule medicines to restore vision.

stem cell science meets small-molecule medicine

stem cells are the regenerative units of our body - they replenish our tissues
constantly, and help fight disease and ageing - they accomplish their task by producing
progenitor cells that give rise to specialized cells through a process called
differentiation - different types of endogenous stem cells reside in the respective
tissues they constantly regenerate – retinal stem cells in the eye, for example.

degenerative diseases are common and becoming more prevalent due to demographic changes -
but these diseases could, in principle, be rescued by stem cells. the opportunity
offered by endogena therapeutics’ approach bears the potential for a dramatic
improvement on existing therapies.

in our lead program, we are targeting degenerative diseases of the eye, including
retinitis pigmentosa, which progressively leads to complete loss of vision – our
pioneering approach enables the discovery of breakthrough therapies based on
cutting-edge knowledge of molecular pathways that regulate retinal stem cells and
retinal pigment epithelial cells, which nourish the eye’s photoreceptors and
retina.

our platform

endogena is building and integrating

a novel, proprietary platform that allows the redefinition of drug discovery, starting
directly from developmental biology - the accurately defined network of pathways
orchestrated by nature to ensure the unobstructed assembly of all multicellular
organisms has divulged its operating modes over the recent decades.

a given compound’s involvement

in a known mode of action is simultaneously tracked and successfully predicted. this
allows for an accurate stem cell lineage specific separation between compound effects on
proliferation and differentiation - collectively, the output from these evaluations is
used to tailor a chemical design strategy customized to a specific target.

collaborations

at endogena, we strongly believe in the innovation potential by collaboration
– accordingly, we work with world-leading partners in academia and industry to leverage
state-of-the-art discoveries and technologies